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Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies

<i>Background and objectives</i>: Choroideremia (CHM) is an X-linked recessive chorioretinal dystrophy caused by mutations involving the <i>CHM</i> gene. Gene therapy has entered late-phase clinical trials, although there have been variable results. This review gives a summary on the outcomes of pha...

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Principais autores: Alessandro Abbouda, Filippo Avogaro, Mariya Moosajee, Enzo Maria Vingolo
Format: Artigo
Sprog:Inglês
Udgivet: MDPI AG 2021-01-01
Serier:Medicina
Fag:
choroideremia
gene
therapy
clinical trials
stem cells
ataluren
Online adgang:https://www.mdpi.com/1648-9144/57/1/64
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